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Clinical outcomes of low-dose pharmacokinetic-guided extended half-life versus low-dose standard half-life factor VIII concentrate prophylaxis in haemophilia A patients.
- Source :
-
Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2023 Jan; Vol. 29 (1), pp. 156-164. Date of Electronic Publication: 2022 Nov 21. - Publication Year :
- 2023
-
Abstract
- Introduction: Despite receiving standard half-life (SHL) factor VIII (FVIII) concentrates prophylaxis, some severe haemophilia A (HA) patients still encounter spontaneous breakthrough bleeding. Individualized pharmacokinetic (PK)-guided dosing of extended half-life (EHL) FVIII concentrates may reduce their bleeding events.<br />Aim: To compare clinical outcomes before and after switching low-dose prophylaxis using weight-based SHL FVIII to PK-guided EHL FVIII concentrates, taking into consideration of a trough FVIII activity at 1 IU/dl above natural baseline.<br />Methods: In this single-centre prospective cohort, Thai severe or moderate HA (FVIII activity ≤3 IU/dl) patients receiving low-dose weight-based SHL FVIII prophylaxis were enrolled. After a 3-day wash-out period, participants underwent low-dose EHL FVIII prophylaxis with PK-based adjustment (myPKFiT <superscript>®</superscript> ) for 6 months. The annualized bleeding rates (ABR), the annualized joint bleeding rates (AJBR), the haemophilia-specific quality-of-life (Haemo-QoL or Haemo-QoL-A) scores, the Hemophilia Joint Health Scores (HJHS) and the annualized FVIII consumption were compared between the two prophylactic periods.<br />Results: Of 15 eligible subjects (mean age 18.7 ± 6.7 years), ABR, AJBR and HJHS were significantly reduced (mean differences of -11.1 ± 4.9 bleeds/year, -10.4 ± 5.2 joint bleeds/year and -5.1 ± 1.5 marks, respectively; P < .001 for all comparisons) after switching regimen. The quality-of-life scores had also improved (P = .001). Nonetheless, FVIII consumption tended to increase despite no statistical significance (means of 1240.9 ± 531.3 SHL FVIII IU/kg/year versus 1591.7 ± 438.9 EHL FVIII IU/kg/year; P = .05).<br />Conclusions: This is the first low-dose, PK-guided, EHL FVIII prophylaxis clinical study in Thailand. Benefits and practicability of this personalized regimen may support the implementation of regular FVIII prophylaxis in developing countries with budget constraints.<br />Clinicaltrials: gov NCT05281185.<br /> (© 2022 John Wiley & Sons Ltd.)
Details
- Language :
- English
- ISSN :
- 1365-2516
- Volume :
- 29
- Issue :
- 1
- Database :
- MEDLINE
- Journal :
- Haemophilia : the official journal of the World Federation of Hemophilia
- Publication Type :
- Academic Journal
- Accession number :
- 36409282
- Full Text :
- https://doi.org/10.1111/hae.14700